A Randomized, Controlled Trial of Ganaxolone in Patients With Infantile Spasms
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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT00441896 |
Recruitment Status :
Completed
First Posted : March 1, 2007
Last Update Posted : November 17, 2020
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Infantile Spasms | Drug: Ganaxolone Other: Placebo | Phase 2 |
Male or female, 4 to 24 months of age (inclusive) with a diagnosis of IS with a 24 hour video EEG (vEEG) recording confirming the diagnosis and previously treated with 3 or fewer antiepileptic drugs (AEDs) are eligible for the study. The subject is able to continue treatment with concomitant AEDs (no more than 2; adrenocorticotropic hormone [ACTH], corticosteroids, felbamate, and vigabatrin are not allowed concomitantly). A ketogenic diet is permitted if it can be maintained for the duration of the study.
There will be a total of three weekly 24-hr video EEGs (baseline, end of weeks 1 and 2 of treatment). Dosing titration begins the day after each video EEG during the inpatient stay. All subjects will be receiving ganaxolone the day after the second video EEG.
A Data Monitoring Board (DMB) will determine whether successive cohorts of subjects can be dosed at an increased dose level; up to a maximum of 6 cohorts.
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 56 participants |
Allocation: | Randomized |
Intervention Model: | Crossover Assignment |
Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
Primary Purpose: | Treatment |
Official Title: | A Double-blind, Placebo-controlled, Dose-ranging Clinical Study to Evaluate the Safety, Tolerability, and Antiepileptic Activity of Ganaxolone in Treatment of Patients With Infantile Spasms |
Study Start Date : | January 2007 |
Actual Primary Completion Date : | May 2008 |
Actual Study Completion Date : | May 2008 |

Arm | Intervention/treatment |
---|---|
Experimental: ganaxolone
ganaxolone
|
Drug: Ganaxolone |
Placebo Comparator: non-active drug
placebo
|
Other: Placebo |
- Spasm frequency as measured by a 24-hour vEEG at Visit 5 (Day 9 ±1 day). [ Time Frame: 1 week ]
- Spasm frequency after two weeks of treatment, as determined by 24-hour vEEG. [ Time Frame: 2 weeks ]
- Cessation of hypsarrhythmia, as determined by vEEG. [ Time Frame: 1 week, 2 weeks ]
- Investigator and caregiver global assessment of seizure severity and response to treatment at Weeks 1 and 2. [ Time Frame: 1 week, 2 weeks ]
- The number of subjects spasm-free and seizure-free (for at least 24 hours) at Weeks 1 and 2. [ Time Frame: 1 week, 2 weeks ]
- Responders (≥ 50% decrease in spasm frequency) at Weeks 1 and 2. [ Time Frame: 1 week, 2 weeks ]
- Reduction of other seizure types at Weeks 1 and 2. Parents/caregivers will maintain a spasm/seizure diary for clinical study subjects. [ Time Frame: 1 week, 2 weeks ]
- Investigators and caregivers assessment of the presence and absence of spasms in each subject following treatment. [ Time Frame: 1 week, 2 weeks ]
- Developmental assessment. [ Time Frame: 2 weeks ]

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 4 Months to 24 Months (Child) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Be diagnosed with IS (regardless of etiology- except for a progressive neurologic illness). Diagnostic Criteria: Seizures consisting of single or repetitive short muscular contractions leading to flexion or extension. Spasms may be characterized as tonic or myoclonic contractions, may occur singly or in clusters, and typically occur bilaterally and symmetrically. The EEG pattern must be consistent with the diagnosis of IS (hypsarrhythmia, modified hypsarrhythmia, multifocal spike wave discharges, etc).
- Have a vEEG recording confirming the diagnosis of IS.
- Have had a magnetic resonance imaging (MRI) performed to determine any possible causes of IS.
- Have been previously treated with 3 or fewer AEDs.
-
If being treated with concomitant AEDs
- Current AEDs have been at a constant daily dose for at least 2 weeks; Note: Subjects with minor dose adjustments may be allowed to enter the study after shorter periods after detailed discussion with the medical monitor.
- Have a stable clinical response/plateau for at least 2 weeks
- Are able to continue treatment with no more than 2 concomitant AEDs (ACTH, corticosteroids, felbamate, and vigabatrin are not allowed concomitantly).
- A ketogenic diet is permitted if it can be maintained for the duration of the study.
- Be a male or female, 4 to 24 months of age (inclusive)
- Have a Parent/Guardian who is properly informed of the nature and risks of the clinical study, who is willing and capable of complying with all clinical study procedures, and has given informed consent in writing prior to entering the clinical study
- Be able to participate for the full term of the clinical study.
Exclusion Criteria:
- Treatment with corticosteroids, ACTH, vigabatrin, felbamate, or any AED not approved by Regulatory Agencies, 2 weeks prior to randomization.
- Treatment with more than two AEDs at baseline.
- Have an active CNS infection, demyelinating disease, degenerative neurological disease, or CNS disease deemed progressive (with the exception of tuberous sclerosis) as evaluated by brain imaging (MRI).
- Have any disease or condition (medical or surgical) at screening that might compromise the hematologic, cardiovascular, pulmonary, renal, gastrointestinal, or hepatic systems; or other conditions that might interfere with the absorption, distribution, metabolism, or excretion of the investigational product, or would place the subject at increased risk.
- Aspartate aminotransferase (AST), alanine aminotransferase (ALT), or total bilirubin greater than four times the upper limit of normal (ULN) or clinical laboratory value deemed clinically significant by the Investigator.
- History of recurrent status epilepticus.
- Have been exposed to any other investigational drug within 30 days prior to randomization.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00441896
United States, California | |
Children's Hospital of Los Angeles | |
Los Angeles, California, United States, 90027 | |
Mattel Children's Hospital at UCLA | |
Los Angeles, California, United States, 90095 | |
United States, District of Columbia | |
Children's National Medical Center | |
Washington, District of Columbia, United States, 20010 | |
United States, Florida | |
Miami Children's Hospital, The Brain Institute | |
Miami, Florida, United States, 33155 | |
Child Neurology Care Center of Northwest Florida | |
Pensacola, Florida, United States, 32504 | |
Child Neurology Center of Northwest Florida | |
Pensacola, Florida, United States | |
United States, Illinois | |
University of Chicago Comer Children's Hospital | |
Chicago, Illinois, United States, 60637 | |
United States, Minnesota | |
Minnesota Epilepsy Group, P.A. | |
Saint Paul, Minnesota, United States, 55102 | |
United States, New York | |
Montefiore Medical Center- Albert Einstein College of Medicine | |
Bronx, New York, United States, 10467 | |
United States, Tennessee | |
Le Bonheur Children's Medical Center | |
Memphis, Tennessee, United States, 38105 | |
United States, Texas | |
Dallas Pediatric Neurology Associates | |
Dallas, Texas, United States, 75230 | |
Texas Children's Hospital | |
Houston, Texas, United States, 77030 | |
United States, Virginia | |
Virginia Commonwealth University Health System | |
Richmond, Virginia, United States, 23298 | |
United States, Washington | |
Children's Hospital and Regional Medical Center | |
Seattle, Washington, United States, 98105 | |
United States, Wisconsin | |
Children's Hospital of Wisconsin | |
Milwaukee, Wisconsin, United States, 53201 |
Study Director: | Joseph Hulihan, MD | Marinus Pharmaceuticals, Inc. |
Responsible Party: | Marinus Pharmaceuticals |
ClinicalTrials.gov Identifier: | NCT00441896 |
Other Study ID Numbers: |
1042-0500 |
First Posted: | March 1, 2007 Key Record Dates |
Last Update Posted: | November 17, 2020 |
Last Verified: | November 2020 |
infantile spasms anticonvulsant pediatric epilepsy West Syndrome epileptic spasms |
Muscle Cramp Spasm Spasms, Infantile Neuromuscular Manifestations Neurologic Manifestations Nervous System Diseases Muscular Diseases Musculoskeletal Diseases Epilepsy, Generalized Epilepsy |
Brain Diseases Central Nervous System Diseases Epileptic Syndromes Ganaxolone Neurosteroids Neurotransmitter Agents Molecular Mechanisms of Pharmacological Action Physiological Effects of Drugs GABA Modulators GABA Agents |